Secondary analyses of dietary data from PRISM clinical trials to evaluate safety and efficacy of pegvaliase for adults with PKU. Public Deposited

Phenylketonuria (PKU) is a genetic disorder caused by a deficiency of phenylalanine hydroxylase leading to decreased conversion of phenylalanine (Phe) to tyrosine (Tyr). Pegvaliase™ (Palynziq™, BioMarin Pharmaceutical Inc., Novato, CA, USA), an injectable enzyme replacement therapy decreases plasma Phe by converting Phe intro trans-cinnamic acid and ammonia. This study aims to evaluate the changes in the dietary composition of participants with PKU on pegvaliase.


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  • khoo.vivien.2019.pdf
  • https://doi.org/10.6083/9w0323552
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  • 2019
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